Summary of Rocket Pharmaceuticals FY Conference Call (January 14, 2026) Company Overview - **Company**: Rocket Pharmaceuticals (NasdaqGM:RCKT) - **Industry**: Biotechnology, specifically focused on gene therapies for rare genetic diseases - **Core Focus**: Development of gene therapies targeting complex and rare genetic diseases, particularly in the cardiovascular space Key Points and Arguments Financial Position - As of September 30, 2025, Rocket reported **$222.8 million** in cash, cash equivalents, and restricted cash, expected to fund operations into the second quarter of **2027** [2] - Anticipated approval of **Kresladi** on March 28, 2026, could yield a **PRV** (Priority Review Voucher), providing strategic non-dilutive capital to extend the cash runway [2] Strategic Focus - 2026 is designated as a year of execution, with a concentrated effort on advancing three cardiovascular programs [2] - The company has deprioritized investments in **Fanconi anemia** to focus on areas with immediate impact [3] Cardiovascular Programs - **Danon Disease**: - A severe X-linked cardiomyopathy with a prevalence of **15,000 to 30,000** in the U.S. and Europe [8] - The lead program shows **100% LAMP2 expression** durability in treated patients, with a median reduction of **24%** in LV mass index over time [9][11] - The program is viewed as a significant near-to-medium-term value driver due to its clear differentiation and market potential [28] - **PKP2 Arrhythmogenic Cardiomyopathy (ACM)**: - Estimated to affect **50,000** adults and children in the U.S. and Europe [17] - The program is currently in phase one, with preliminary safety and efficacy results indicating well-tolerated gene therapy [18] - The company aims to define a registrational pathway for this program in 2026 [20] - **BAG3 Dilated Cardiomyopathy (DCM)**: - Considered a potential largest market opportunity with around **30,000** individuals affected in the U.S. [24] - The program is in the process of initiating a phase one trial, focusing on safety and preliminary efficacy [25] Regulatory and Clinical Development - The company is working towards resuming patient dosing for Danon disease, with logistics being the primary gating factor [29] - A concurrent natural history study for Danon is ongoing, validating the rapid decline of untreated patients [35] - The regulatory review for Kresladi is mid-cycle, with no current showstoppers noted [37] Manufacturing Capabilities - Rocket has in-house manufacturing capabilities for AAV, which helps control costs and timelines [38] - The facility is designed to support current and future programs, ensuring high margins and process improvements over time [38] Future Outlook - The anticipated approval of Kresladi is seen as a pivotal moment for transitioning to a commercial-stage gene therapy company [26] - The company plans to maintain a steady pipeline with additional programs ready to advance in the future [40] Additional Important Insights - The company emphasizes the importance of achieving meaningful myocardial transduction and protein localization for clinical benefit [21] - There is a strong community interest in the Danon program, despite past challenges, indicating a significant unmet medical need [32] - The company is cautious but optimistic about the regulatory landscape and the potential for future growth [37] This summary encapsulates the key points discussed during the conference call, highlighting Rocket Pharmaceuticals' strategic focus, financial position, and advancements in their gene therapy programs.

Core Insights - Rocket Pharmaceuticals (NASDAQ:RCKT) saw an approximate 11% increase in premarket trading following the announcement that the FDA accepted its resubmitted marketing application for the gene therapy candidate Kresladi [4] - The FDA had previously rejected the company's biologics license application in June 2024 [4]

Core Insights - Rocket Pharmaceuticals (RCKT) has received FDA clearance for its investigational new drug application to initiate clinical studies for its gene therapy candidate RP-A701, aimed at treating BAG3-associated dilated cardiomyopathy [1][6] - The planned phase I study will assess the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM, a rare genetic heart condition [2][6] Company Pipeline - RCKT is also developing another gene therapy candidate, RP-A601, which is in early-stage development for arrhythmogenic cardiomyopathy [3] - Recent setbacks in the company's pipeline, including a voluntary pause in the phase II study of RP-A501 due to a patient death, have raised concerns [4][7] - The FDA has issued a complete response letter regarding the biologics license application for Kresladi, requesting additional information [8] Stock Performance - Year to date, RCKT's shares have declined by 80.5%, contrasting with a 3.6% decline in the industry [3] - The company currently holds a Zacks Rank of 3 (Hold), while other biotech stocks like Exelixis, Spero Therapeutics, and Puma Biotechnology have better rankings [9]

Core Viewpoint - Rocket Pharmaceuticals (RCKT) shares fell 62.8% following the announcement of a patient death in a pivotal phase II study for its gene therapy candidate RP-A501, aimed at treating Danon disease [1][2] Company Developments - RCKT has voluntarily paused further dosing in the RP-A501 study after the FDA placed a clinical hold on it [2] - The company is conducting a comprehensive root cause analysis to understand the serious adverse event that led to the patient's death [2] - RCKT is collaborating with the FDA, Independent Data Safety Monitoring Committee, clinical investigators, and scientific experts to ensure patient safety and to restart the study as soon as possible [3] Stock Performance - Year-to-date, RCKT shares have declined 81.5%, significantly underperforming the industry, which has seen a decline of 5.8% [4] Pipeline Setbacks - RCKT has faced setbacks with its pipeline candidates, particularly with Kresladi, which is intended to treat severe leukocyte adhesion deficiency-I (LAD-I) [5] - The FDA issued a complete response letter (CRL) regarding the biologics license application (BLA) for Kresladi, requesting limited additional information on Chemistry Manufacturing and Controls (CMC) [7] - This marks the second request for additional information from the FDA regarding Kresladi's CMC, with the review period extended by three months in February 2024 [7] - The company plans to file a complete BLA to address the CRL later in 2025 [8]

Financial Performance - Rocket Pharmaceuticals incurred a loss of 56 cents per share in Q1 2025, which is narrower than the Zacks Consensus Estimate of a loss of 59 cents and an improvement from a loss of 66 cents per share in the same quarter last year [1] - The company did not record any revenues in the reported quarter, missing the Zacks Consensus Estimate for total revenues of $8 million [2] - General and administrative expenses rose 28% year over year to $28.4 million, attributed to increased commercial preparation and legal expenses [2] - Research and development expenses were $35.9 million, down 21% from the previous year due to reduced manufacturing and development costs [3] - As of March 31, 2025, the company had cash, cash equivalents, and investments totaling $318.2 million, down from $372.3 million as of December 31, 2024, with expectations to fund operations into Q4 2026 [3] - Year to date, RCKT shares have declined 44%, compared to an 8% decline in the industry [4] Pipeline Developments - Kresladi, developed for treating severe leukocyte adhesion deficiency-I (LAD-I), received a complete response letter (CRL) from the FDA in June 2024, requesting limited additional information on the Chemistry Manufacturing and Controls (CMC) [7][8] - The company plans to file a complete BLA to resolve the CRL later in 2025 [8] - Rocket Pharmaceuticals is developing RP-L102 for treating Fanconi anemia (FA) and has initiated a rolling BLA, expecting to complete the submission in late 2025 or early 2026 [9] - Dosing is currently underway in a phase II pivotal study for RP-A501, targeting male patients with Danon disease, with clinical data readout expected in mid-2026 [10]

Core Viewpoint - Rocket Pharmaceuticals reported a narrower loss per share in Q4 2024 compared to estimates, but did not generate any revenue due to the absence of marketed products in its portfolio [1][2][7]. Financial Performance - In Q4 2024, Rocket Pharmaceuticals incurred a loss of $0.62 per share, which was better than the Zacks Consensus Estimate of a loss of $0.71 per share and a loss of $0.64 per share in the same quarter last year [1]. - For the full year 2024, the company reported a loss of $2.73 per share, an improvement from a loss of $2.92 in 2023 [7]. - The company did not record any revenues in both Q4 2024 and the full year 2024 [2][7]. Expenses - General and administrative expenses increased by 17.7% year over year to $25.3 million, attributed to higher commercial preparation expenses [3]. - Research and development expenses were $37.4 million, down 10.3% from the previous year, due to reduced manufacturing and development costs [3]. Cash Position - As of December 31, 2024, Rocket Pharmaceuticals had cash, cash equivalents, and investments totaling $372.3 million, up from $235.7 million on September 30, 2024 [4]. - Management anticipates that this cash balance will support operations and capital expenditures through the third quarter of 2026 [4]. Pipeline Developments - The company is developing Kresladi for treating severe leukocyte adhesion deficiency-I (LAD-I), but received a complete response letter from the FDA in June 2024, requesting additional information [8][9]. - Rocket Pharmaceuticals has initiated a rolling biologics license application (BLA) for RP-L102, aimed at treating Fanconi anemia, which is also under review in the EU [10]. - Dosing is ongoing in a phase II study for RP-A501, targeting Danon disease, with updates expected in the first half of 2025 [11]. - RP-A601, another AAV-based gene therapy candidate, is in phase I development for arrhythmogenic cardiomyopathy, with initial data anticipated in the first half of 2025 [12].